tofersen: Clinical Trials

3 completed, 2 active not recruiting, 2 recruiting, 1 approved for marketing. 3 Phase 3, 1 Phase 1, 1 Phase 2, 1 Phase 4.

By phase and status

Phase	Trials	Status
Phase 3	3	Completed 2, Active not recruiting 1
Phase 1	1	Completed 1
Phase 2	1	Recruiting 1
Phase 4	1	Active not recruiting 1

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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS) Tofersen - Amyotrophic Lateral Sclerosis		Recruiting	Biogen	125	2026-01-02	2026-04-09
Tofersen in Non-SOD1 ALS Tofersen - ALS (Amyotrophic Lateral Sclerosis)	Phase 2	Recruiting	Washington University School of Medicine	30	2025-12-29	2026-06-25
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS) Tofersen - Amyotrophic Lateral Sclerosis	Phase 4	Active not recruiting	Biogen	12	2025-12-11	2026-08-13
Study to Learn If Tofersen (BIIB067) Can Delay ALS Symptoms and About Its Safety in Adults With a SOD1 Gene Mutation Who Have Not Yet Developed ALS Symptoms Tofersen, Placebo - Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation	Phase 3	Active not recruiting	Biogen	158	2021-05-17	2026-11-04
A Study to Evaluate Safety, Tolerability, and Distribution of a Microdose of Radiolabeled BIIB067 (99mTc-MAG3-BIIB067) Co-administered With BIIB067 (Tofersen) to Healthy Adults Tofersen, 99mTc-MAG3-BIIB067 - Healthy Volunteers	Phase 1	Completed	Biogen	8	2018-12-20	2023-06-22
Long-Term Evaluation of BIIB067 (Tofersen) Tofersen - ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation	Phase 3	Completed	Biogen	139	2017-03-08	2025-08-29
An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS) Tofersen, Placebo - Amyotrophic Lateral Sclerosis	Phase 3	Completed	Biogen	176	2016-01-20	2023-07-28
Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis Tofersen - Superoxide Dismutase 1-Amyotropic Lateral Sclerosis		Approved for marketing	Biogen			2024-06-10

Conditions studied

Amyotrophic Lateral Sclerosis (4)

Sponsors

Biogen (7)  ·  Washington University School of Medicine (1)

Countries

United States (7)  ·  Belgium (3)  ·  Canada (3)  ·  France (3)  ·  Germany (3)  ·  Italy (3)  ·  Japan (3)  ·  United Kingdom (3)  ·  Australia (2)  ·  Denmark (2)  ·  Poland (2)  ·  South Korea (2)  ·  Brazil (1)  ·  China (1)  ·  Spain (1)

Results publications

PMID 36129998, PMID 41661214, PMID 32640130, PMID 35585374, PMID 41233143, PMID 42406382

NIH funding

2015 (1), 2016 (1), 2023 (1)